NCT02675465CompletedPhase 1/2
First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221
An Open-Label, Fixed-Sequence, Ascending-Dose, First-in-Human Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of Intravenous Infusions of ATB200 Co-Administered With Oral AT2221 in Adult Subjects With Pompe Disease
Source: ClinicalTrials.gov · Last updated 23 Oct 2025
Brief summary (registry, in English)
This is an international, multi-center, open-label study designed to evaluate if the co-administration of investigational new drugs ATB200 and AT2221 is safe in adults with Pompe disease.
- Status (registry)
- Completed
- Phase (registry)
- Phase 1/2
- Study type
- Interventional
- Enrollment
- 29 (actual)
- First submitted
- 26 Jan 2016
- Start date
- 1 Apr 2016
- Primary completion date
- 22 Aug 2024
- Lead sponsor
- Amicus Therapeutics
- Diseases
- Pompe disease
- NCT number
- NCT02675465
What you commit to
- Study type: Interventional · Phase 1/2
- Participants enrolled: 29
- From 1 Apr 2016 to the primary completion date, 22 Aug 2024
- 19 sites in 6 countries
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Received treatment with prohibited medications within 30 days of Baseline Visit
Other illness
- Had a medical or any other extenuating condition or circumstance that may, in opinion of investigator, pose an undue safety risk to the subject or compromise his/her ability to comply with protocol requirements
- Had a history of allergy or sensitivity to alglucosidase alfa, miglustat or other iminosugars (Cohorts 1, 2, and 4)
- Had active systemic autoimmune disease such as lupus, scleroderma, or rheumatoid arthritis; subjects with autoimmune disease must have been discussed with the Amicus Medical Monitor
Other
- Subject, if female, was pregnant or breastfeeding at screening
- Subject, whether male or female, planned to conceive a child during the study
- Required invasive ventilatory support, or used noninvasive ventilatory support ≥ 6 hours a day while awake (Cohorts 1, 3, and 4)
- Had active bronchial asthma; subjects with bronchial asthma must have been discussed with the Amicus Medical Monitor
Criteria the registry did not label
- Adults with Diagnosis of Pompe disease
- Cohort 1: Enzyme Replacement Therapy (ERT)-experienced subject (ambulatory):
- Male and female subjects between 18 and 65 years of age, inclusive
- Received ERT with alglucosidase alfa (Myozyme/Lumizyme) for the previous 2-6 years, inclusive
- Was receiving alglucosidase alfa at a frequency of once every other week
- Must have been able to walk 200-500 meters on the 6-Minute Walk Test (6MWT)
- Had upright Forced Vial Capacity (FVC) 30-80% of predicted normal value
- Cohort 2: ERT-experienced subjects (non-ambulatory):
- Male and female subjects between 18 and 65 years of age, inclusive
- Had been receiving ERT with alglucosidase alfa for ≥2 years at a regular or set frequency
- Was wheelchair-bound
- Cohort 3: ERT-naïve subjects (ambulatory):
- Male and female subjects between 18 and 65 years of age, inclusive
- Must have been able to walk 200-500 meters on the 6MWT
- Had upright FVC 30-80% of predicted normal value
- Cohort 4: ERT-experienced subject (ambulatory):
- Male and female subjects between 18 and 75 years of age, inclusive
- Had been receiving ERT with alglucosidase alfa for ≥7 years, inclusive
- Was receiving alglucosidase alfa at a frequency of once every other week
- Must have been able to walk 75-600 meters on the 6MWT
- Had upright FVC 30-85% of predicted normal value
Age in the registry: 18 years to 75 years · Sex: All · Does not accept healthy volunteers
Sites (19)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Womens & Childrens Hospital, Adelaide Coordinator of this site? Confirm its status | North Adelaide | Australia | Unknown status |
| University Children's Hospital Department of Neuropediatrics and Inborn Metabolic Disorders, St. Josefs-Hospital Coordinator of this site? Confirm its status | Bochum | Germany | Unknown status |
| Friedrich-Baur-Institure, Dep of Neurology - University Munich Coordinator of this site? Confirm its status | Munich | Germany | Unknown status |
| University Hospital Birmingham NHS Foundation Trust, Queen Elizabeth Medical Center Coordinator of this site? Confirm its status | Birmingham | United Kingdom | Unknown status |
| Salford Royal NHS Foundation Trust Coordinator of this site? Confirm its status | Salford | United Kingdom | Unknown status |
| Erasmus Medical Center Coordinator of this site? Confirm its status | Rotterdam | Netherlands | Unknown status |
| School of Medicine, University of Auckland Coordinator of this site? Confirm its status | Auckland | New Zealand | Unknown status |
| Emory University Division of Medical Genetics Coordinator of this site? Confirm its status | Decatur | United States | Unknown status |
| Duke University Medical Center Coordinator of this site? Confirm its status | Durham | United States | Unknown status |
| Lysosomal & Rare Disorders Research & Treatment Center (LDRTC) Coordinator of this site? Confirm its status | Fairfax | United States | Unknown status |
| University of Florida Coordinator of this site? Confirm its status | Gainesville | United States | Unknown status |
| Infusion Associates Coordinator of this site? Confirm its status | Grand Rapids | United States | Unknown status |
| Great Falls Clinic LLP Coordinator of this site? Confirm its status | Great Falls | United States | Unknown status |
| Rutgers New Jersey Medical School Coordinator of this site? Confirm its status | Newark | United States | Unknown status |
| University of California Irvine Coordinator of this site? Confirm its status | Orange | United States | Unknown status |
| Perelman Center for Advanced Medicine Coordinator of this site? Confirm its status | Philadelphia | United States | Unknown status |
| Neuromuscular Research Centre Coordinator of this site? Confirm its status | Phoenix | United States | Unknown status |
| University of Pittsburgh Coordinator of this site? Confirm its status | Pittsburgh | United States | Unknown status |
| Abramson Cancer Center Chester County Hospital Coordinator of this site? Confirm its status | West Chester | United States | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.